SAN DIEGO, August 13, 2026

Capricor Therapeutics continues to advance Deramiocel (CAP-1002) for Duchenne muscular dystrophy (DMD) as the company works with the U.S. Food and Drug Administration on the ongoing review of its Biologics License Application (BLA). Deramiocel is Capricor’s lead allogeneic cell therapy candidate and is being developed to address the progressive skeletal and cardiac muscle complications associated with DMD. The regulatory review follows the completion of the Phase 3 HOPE-3 study, in which Deramiocel achieved its primary endpoint related to upper limb function. The full HOPE-3 dataset was subsequently published in The Lancet, providing independently peer-reviewed evidence for the therapy’s clinical performance. Capricor said its priority remains advancing Deramiocel toward patients and families affected by DMD while continuing discussions with the FDA regarding a potential regulatory pathway. The company is also preparing for additional regulatory and commercial activities as the FDA review progresses.

HOPE-3 Data Support Deramiocel Development

The HOPE-3 Phase 3 trial remains a central component of Capricor’s regulatory strategy for Deramiocel. The study demonstrated a statistically significant slowing of upper limb disease progression, measured using the PUL 2.0 assessment, with a primary endpoint result of p=0.029. Additional analyses provided supportive findings across functional and cardiac measures. Following publication of the complete dataset, Capricor clarified a statistical-model issue identified during the peer-review process and reverted to the statistical analysis plan established before unblinding. The company said the change affected the left ventricular ejection fraction analysis but did not affect the HOPE-3 primary endpoint, while the prespecified cardiomyopathy subgroup remained unchanged. Deramiocel has also accumulated substantial clinical experience, with approximately 1,300 intravenous infusions administered to more than 200 DMD patients across three clinical trials. More than 80 patients remain enrolled in open-label extension studies, with some receiving treatment for more than five years, supporting continued evaluation of the therapy’s long-term safety and durability.

FDA Review and Manufacturing Readiness Continue

Capricor’s Deramiocel BLA remains under active FDA review, with the company continuing regulatory interactions and expecting to provide further updates as the review advances. On July 29, 2026, the FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted 3 in favor and 9 against whether the available evidence provided substantial evidence of effectiveness for Deramiocel in DMD-associated cardiomyopathy. The committee’s recommendation is advisory and non-binding, and Capricor noted that the committee was not asked to vote on the HOPE-3 primary endpoint or the overall benefit-risk profile. Separately, the FDA conducted a Bioresearch Monitoring inspection in July and issued a Form 483 containing one observation; Capricor has submitted its response and is awaiting further feedback. On the manufacturing side, the company’s GMP facility in San Diego is operational and positioned to support an initial commercial launch if Deramiocel receives approval. Capricor is also advancing commercial preparations while maintaining a measured approach as regulatory clarity develops.

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Financial Position Supports Deramiocel Program

Capricor reported approximately $237.9 million in cash, cash equivalents and marketable securities as of June 30, 2026, compared with approximately $318.1 million at the end of 2025. The company recorded no revenue during the first half of 2026, while second-quarter operating expenses increased to approximately $42.9 million and net loss reached approximately $40.7 million. Capricor said its available financial resources are expected to fund operating capital requirements for at least the next twelve months under its current plan. Beyond the U.S. regulatory process, the company has initiated regulatory engagement in Europe and Japan for Deramiocel and continues to consider future development in younger DMD patients and Becker muscular dystrophy. Other programs, including Capricor’s exosome-based platform, remain on hold pending greater regulatory clarity, keeping Deramiocel and its FDA review as the company’s primary near-term focus.

Source:Capricor Therapeutics,, press relese